European Commission Grants Orphan Drug Designation to Soliris® (eculizumab) for the Treatment of Patients with Myasthenia Gravis (MG)

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CHESHIRE, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals (NASDAQ:ALXN) today announced that the European
Commission has granted orphan drug designation (ODD) to Soliris®
(eculizumab) for the treatment of patients with Myasthenia Gravis (MG),
a rare, debilitating neurologic disorder caused by uncontrolled
complement activation. In patients with MG, uncontrolled complement
activation due to antibodies directed at the neuromuscular junction can
ultimately lead to profound and debilitating weakness of various muscle
groups throughout the body.

“Patients with MG develop debilitating muscle weakness, impairing their
ability to walk, speak clearly, swallow and, in some cases, to breathe
normally, which could lead to a life-threatening myasthenic crisis,”
said Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D
at Alexion. “By specifically inhibiting the terminal complement pathway,
which is believed to play a pivotal role in the pathophysiology of MG,
we believe that eculizumab has the potential to help patients living
with this devastating rare disorder.”

Soliris is a first-in-class terminal complement inhibitor and is
currently approved for the treatment of patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome
(aHUS), two debilitating, ultra-rare and life-threatening disorders
caused by chronic uncontrolled complement activation. Soliris is not
approved in any country to treat MG. Alexion is enrolling patients in a
multinational, placebo-controlled registration trial of eculizumab in
patients with refractory generalized MG. More information on this trial
is available at www.clinicaltrials.gov
under the identifier NCT01997229.

The European Commission grants orphan medicinal product status to
provide incentives to develop medicinal products to treat, prevent or
diagnose diseases or conditions that affect no more than five in 10,000
persons in the EU. The orphan medicinal product status designation would
provide Alexion with certain benefits and incentives in the EU,
including a period of market exclusivity if Soliris is ultimately
approved for the designated indication.

About Myasthenia Gravis (MG)

Myasthenia Gravis is a rare, debilitating neurologic disorder caused by
auto-antibodies that recognize a specific target in the nerve-muscle
junction, which results in life-long uncontrolled terminal complement
activation causing tissue damage and interference with signalling
between nerve and muscle fibers.1,2 Patients with MG
initially experience weakness in their ocular (eye) muscles, and the
disease typically progresses to the more severe and generalized form to
include weakness of head, trunk, limb and respiratory muscles. Symptoms
can include drooping eyelid, weakness in the arms and legs, slurred
speech, difficulty chewing or swallowing, and difficulty breathing,
which could lead to a life-threatening myasthenic crisis.

About Soliris

Soliris is a first-in-class terminal complement inhibitor developed from
the laboratory through regulatory approval and commercialization by
Alexion. Soliris is approved in the U.S. (2007), European Union (2007),
Japan (2010) and other countries as the first and only treatment for
patients with paroxysmal nocturnal hemoglobinuria (PNH), a debilitating,
ultra-rare and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells). Soliris
is indicated to reduce hemolysis. Soliris is also approved in the U.S.
(2011), the European Union (2011), Japan (2013) and other countries as
the first and only treatment for patients with atypical hemolytic uremic
syndrome (aHUS), a debilitating, ultra-rare and life-threatening genetic
disorder characterized by complement-mediated thrombotic
microangiopathy, or TMA (blood clots in small vessels). Soliris is
indicated to inhibit complement-mediated TMA. Soliris is not indicated
for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). For the breakthrough innovation in
complement inhibition, Alexion and Soliris have received the
pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award
for Best Biotechnology Product with broad implications for future
biomedical research and the 2009 Prix Galien France Award in the
category of Drugs for Rare Diseases.

The U.S. product label for Soliris includes a boxed warning:
"Life-threatening and fatal meningococcal infections have occurred in
patients treated with Soliris. Meningococcal infection may become
rapidly life-threatening or fatal if not recognized and treated early
[see Warnings and Precautions (5.1)]. Comply with the most current
Advisory Committee on Immunization Practices (ACIP) recommendations for
meningococcal vaccination in patients with complement deficiencies.
Immunize patients with a meningococcal vaccine at least two weeks prior
to administering the first dose of Soliris, unless the risks of delaying
Soliris therapy outweigh the risk of developing a meningococcal
infection. [See Warnings and Precautions (5.1) for additional guidance
on the management of the risk of meningococcal infection]. Monitor
patients for early signs of meningococcal infections and evaluate
immediately if infection is suspected. Soliris is available only through
a restricted program under a Risk Evaluation and Mitigation Strategy
(REMS). Under the Soliris REMS, prescribers must enroll in the program
[see Warnings and Precautions (5.2)]. Enrollment in the Soliris REMS
program and additional information are available by telephone:
1-888-SOLIRIS (1-888-765-4747)."

In patients with PNH, the most frequently reported adverse events
observed with Soliris treatment in clinical studies were headache,
nasopharyngitis (runny nose), back pain and nausea. Soliris treatment of
patients with PNH should not alter anticoagulant management because the
effect of withdrawal of anticoagulant therapy during Soliris treatment
has not been established. In patients with aHUS, the most frequently
reported adverse events observed with Soliris treatment in clinical
studies were headache, diarrhea, hypertension, upper respiratory
infection, abdominal pain, vomiting, nasopharyngitis, anemia, cough,
peripheral edema, nausea, urinary tract infections, pyrexia. Soliris is
not indicated for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). Please see full prescribing
information for Soliris, including BOXED WARNING regarding risk of
serious meningococcal infection.

About Alexion

Alexion is a biopharmaceutical company focused on serving patients with
severe and rare disorders through the innovation, development and
commercialization of life-transforming therapeutic products. Alexion is
the global leader in complement inhibition and has developed and markets
Soliris® (eculizumab) as a treatment for patients with PNH and aHUS, two
debilitating, ultra-rare and life-threatening disorders caused by
chronic uncontrolled complement activation. Soliris is currently
approved in nearly 50 countries for the treatment of PNH and in nearly
40 countries for the treatment of aHUS. Alexion is evaluating other
potential indications for Soliris in additional severe and ultra-rare
disorders beyond PNH and aHUS, and is developing other highly innovative
biotechnology product candidates, including asfotase alfa, across
multiple therapeutic areas. This press release and further information
about Alexion can be found at: www.alexionpharma.com.

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Safe Harbor Statement

This news release contains forward-looking statements, including
statements related to potential medical benefits of Soliris®
(eculizumab) for the treatment of patients with Myasthenia Gravis (MG).
Forward-looking statements are subject to factors that may cause
Alexion's results and plans to differ from those expected, including,
for example, decisions of regulatory authorities regarding marketing
approval or material limitations on the marketing of Soliris for MG,
delays in arranging satisfactory manufacturing capabilities and
establishing commercial infrastructure for Soliris for MG, the
possibility that results of clinical trials are not predictive of safety
and efficacy results of Soliris for MG in broader or different patient
populations, the risk that third party payors (including governmental
agencies) will not reimburse for the use of Soliris for MG (if approved)
at acceptable rates or at all, the risk that estimates regarding the
number of patients with Soliris for MG and observations regarding the
natural history of patients with Soliris for MG are inaccurate, and a
variety of other risks set forth from time to time in Alexion's filings
with the Securities and Exchange Commission, including but not limited
to the risks discussed in Alexion's Quarterly Report on Form 10-Q for
the period ended June 30, 2014. Alexion does not intend to update any of
these forward-looking statements to reflect events or circumstances
after the date hereof, except when a duty arises under law.